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New England Journal of Medicine (NEJM) Publishes Pivotal Phase 3 Data...
2015-09-10 06:45:57| Biotech - Topix.net
Alexion Pharmaceuticals, Inc. announced today that data from the pivotal Phase 3 ARISE study evaluating the safety and efficacy of Kanumaa in children and adults with lysosomal acid lipase deficiency have been published in the September 10 issue of the New England Journal of Medicine . In the study, Kanuma met the primary endpoint of alanine aminotransferase normalization compared with placebo as well as six secondary endpoints.1 Kanuma is an innovative enzyme replacement therapy that has been approved by the European Commission for the treatment of patients of all ages with LAL-D, a genetic, chronic, and progressive ultra-rare metabolic disease in which infants, children and adults suffer multi-organ damage and premature death.
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Category:Biotechnology and Pharmaceuticals